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PERSPECTIVES
and the cost that we need. Charles River has a lot of different
components that allow us to work with them on many
different fronts. I expect we'll continue to do so. As long as
quality and costs are in line with our needs, there's no reason
we wouldn't consider Charles River for future work.
BEN MOORE:
We are a patient-centric organization. That's the goal of our
team from top to bottom. Our key considerations are about
quality as it informs us on safety: how can we keep patients
safe, how can we deliver this effectively to them, and how we
can do so with the highest quality.
Anjali Sarkar: Any final thoughts from anyone
before we wrap up?
ERIC SCHAEFFER:
Working with Charles River, we've built a good preclinical
story that makes us believe that the therapy we're developing
should work. Gene therapy has its own challenges, particularly
getting into the brain. That's work that we're starting
to do now in primates initially, to try to understand the right
route and the process of getting expression in the parts of the
brain where we need it.
ADAD affects young people, and it's a devastating
prognosis. Speed is of the essence. These are not patients
who can wait. The quicker we can get to the clinic and
prove to ourselves and others that this is an effective
treatment for ADAD, the more patients we will be able to
help. The day we reach that goal will be a day to break
open the champagne.
16 | GENengnews.com
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ChasRiver_Aug2022_Perspectives-Toward-a-Treatment

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